Tisagenlecleucel demonstrates comparable safety and efficacy in the real-world and clinical trial settings for patients with acute lymphoblastic leukemia (ALL) and non-Hodgkin lymphoma (NHL), according to results published in Blood Advances (2020;4[21]:5414-5424. doi:10.1182/bloodadvances.2020003092).
Tisagenlecleucel is a CD19 chimeric antigen receptor (CAR) T-cell therapy approved for the treatment of pediatric and young adult patients with relapsed/refractory ALL and adults with NHL.
“A better understanding of the safety and efficacy outcomes of patients receiving tisagenlecleucel outside of a clinical trial remains crucial to the care of these patients,” explained Marcelo C Pasquini, MD, Department of Medicine, Center for International Blood and Marrow Transplant Research (CIBMTR), Medical College of Wisconsin, Milwaukee, and colleagues.
This study explored early safety and efficacy outcomes among patients receiving commercial tisagenlecleucel in the real-world setting.
As of January 2020, a total of 511 patients from 73 centers were enrolled in the study. Follow-up data were available for 410 patients (ALL, n = 255; NHL, n = 155), with a median follow-up of 13.4 months for ALL and 11.9 months for NHL.
Among patients with ALL, the initial complete remission (CR) rate was 85.5%. The 12-month duration of response (DOR) was 60.9%, event-free survival was 52.4%, and overall survival (OS) was 77.2%.
Among patients with NHL, the best overall response rate was 61.8%, including an initial CR rate of 39.5%. the 6-month DOR, progression-free survival, and OS rates were 55.3%, 38.7%, and 70.7%, respectively.
Grade ≥3 cytokine release syndrome and neurotoxicity were reported in 11.6% and 7.5% of all patients, respectively.
“This report demonstrates that in the real-world setting, efficacy of tisagenlecleucel for ALL and NHL are comparable to that in the pivotal ELIANA and JULIET trials,” concluded Dr Pasquini and colleagues.—Janelle Bradley
